early treatment

Standardized SMA newborn screening may aid early treatment

Substantial differences in newborn screening (NBS) practices for spinal muscular atrophy (SMA) across the U.S. could impact referral patterns or the timing of therapeutic interventions, a study shows. While healthcare providers evaluated newborns with positive results within the first week of life, many didn’t initiate therapy until after they…

Early SMA diagnosis, treatment a ‘game changer’ in region of Italy

An early spinal muscular atrophy (SMA) diagnosis with the help of a newborn screening program, and treatment with disease-modifying therapies before symptom onset, may prevent the development of SMA symptoms, according to a new study in Italy. The study found that children born without symptoms who started treatment…

Wait times for SMA specialists found shorter than for neurologists

Infants with suspected spinal muscular atrophy (SMA) who are directly referred to an SMA care center may experience shorter wait times than those referred to general neurology practices, according to a survey of these specialists deployed by Cure SMA. While both types of practices reported using triage…

Early treatment may not resolve swallowing problems in SMA: Study

Children with spinal muscular atrophy (SMA) can still experience swallowing difficulties despite early treatment with disease-modifying therapies, a small study shows. Swallowing was either incomplete, with liquid remaining in the mouth and/or esophagus, and/or unsafe due to aspiration (the inhalation of liquid into the lungs), in all of the…